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MechanismCYP21A2 gene transference |
Active Org.- |
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Active Indication- |
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Drug Highest PhaseDiscontinued |
First Approval Ctry. / Loc.- |
First Approval Date- |
/ Active, not recruitingPhase 1/2 A Phase 1/2, First-in-Human, Open-Label, Dose-Escalation Study of the Safety and Efficacy of Gene Therapy for Congenital Adrenal Hyperplasia Through Administration of an Adeno-Associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
This study is designed to evaluate the safety, tolerability, and efficacy of AAV5 based BBP-631 in adult participants diagnosed with classic congenital adrenal hyperplasia.
/ CompletedNot Applicable Pre-screening Study to Identify Adult Participants with Classic Congenital Adrenal Hyperplasia Due to 21-Hydroxylase Deficiency Who May Be Eligible for Treatment in the CAH-301 Trial with BBP-631, an Adeno-associated Virus (AAV) Serotype 5-Based Recombinant Vector Encoding the Human CYP21A2 Gene
This pre-screening study is designed to determine potential eligibility of adults with classic CAH due to 21-hydroxylase deficiency (21-OHD) for participation in the CAH-301 [NCT04783181] gene therapy trial with BBP-631.
100 Clinical Results associated with Adrenas Therapeutics, Inc.
0 Patents (Medical) associated with Adrenas Therapeutics, Inc.
100 Deals associated with Adrenas Therapeutics, Inc.
100 Translational Medicine associated with Adrenas Therapeutics, Inc.